OBJECTIVE: To develop response criteria for juvenile dermatomyositis (DM). METHODS: We analyzed the performance of 312 definitions that used core set measures from either the International Myositis Assessment and Clinical Studies Group (IMACS) or the Paediatric Rheumatology International Trials Organisation (PRINTO) and were derived from natural history data and a conjoint analysis survey. They were further validated using data from the PRINTO trial of prednisone alone compared to prednisone with methotrexate or cyclosporine and the Rituximab in Myositis (RIM) trial. At a consensus conference, experts considered 14 top candidate criteria based on their performance characteristics and clinical face validity, using nominal group technique. RESULTS: Consensus was reached for a conjoint analysis-based continuous model with a total improvement score of 0-100, using absolute percent change in core set measures of minimal (≥30), moderate (≥45), and major (≥70) improvement. The same criteria were chosen for adult DM/polymyositis, with differing thresholds for improvement. The sensitivity and specificity were 89% and 91-98% for minimal improvement, 92-94% and 94-99% for moderate improvement, and 91-98% and 85-86% for major improvement, respectively, in juvenile DM patient cohorts using the IMACS and PRINTO core set measures. These criteria were validated in the PRINTO trial for differentiating between treatment arms for minimal and moderate improvement (P = 0.009-0.057) and in the RIM trial for significantly differentiating the physician's rating for improvement (P < 0.006). CONCLUSION: The response criteria for juvenile DM consisted of a conjoint analysis-based model using a continuous improvement score based on absolute percent change in core set measures, with thresholds for minimal, moderate, and major improvement.

Charles University Prague Czech Republic

Duke University Durham North Carolina

Emory University School of Medicine Atlanta Georgia

Great Ormond Street Hospital for Children NHS Trust London UK

Hannover Medical School Hannover Germany

Hospital de Niños Pedro de Elizalde University of Buenos Aires Buenos Aires Argentina

Hospital de Pediatría Garrahan Buenos Aires Argentina

Istituto Giannina Gaslini Pediatria 2 Reumatologia and Università degli Studi di Genova Genoa Italy

Istituto Giannina Gaslini Pediatria 2 Reumatologia PRINTO Genoa Italy

Istituto Giannina Gaslini Servizio di Epidemiologia e Biostatistica Genoa Italy

IWK Health Centre Halifax Nova Scotia Canada

Karolinska University Hospital Stockholm Sweden

NIH Bethesda Maryland

Royal Hospital for Sick Children Glasgow UK and Royal Hospital for Sick Children Edinburgh UK

Semmelweis University Budapest Hungary

Social and Scientific Systems Inc Durham North Carolina

The Hospital for Sick Children Toronto Ontario Canada

Universidade Estadual Paulista Júlio de Mesquita Filho Botucatu Saõ Paulo Brazil

Universidade Federal do Rio de Janeiro Rio de Janeiro Brazil

University Medical Centre Utrecht Wilhelmina Children's Hospital Utrecht The Netherlands

University of Firenze Florence Italy

University of Kansas City Medical Center Kansas City Kansas

University of Pittsburgh Pittsburgh Pennsylvania

University of Texas Southwestern Medical Center Dallas

Arthritis Rheumatol. 2018 Mar;70(3):467. doi: 10.1002/art.40450. PubMed

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